BRUSSELS, Belgium: U.S. drugmakers are charging significantly more for new treatments, particularly those targeting rare diseases, as the median launch price for new pharmaceuticals more than doubled in just four years, a Reuters analysis has found.

According to a survey of 45 new medicines launched in 2024, the median annual list price exceeded US$370,000 — a sharp rise from $180,000 in 2021. The increase reflects the growing focus on rare diseases, where smaller patient populations often justify higher prices and where scientific advances have made new drug development more feasible.

That trend is accelerating even as the U.S. government takes steps to reduce prescription costs. Drug pricing has become a major political issue, with President Donald Trump calling for prices to better align with what other high-income countries pay.

William Padula, a pharmaceutical and health economics professor at the University of Southern California, said prices are likely to continue rising until the cost of developing therapies can be brought down. "For rare diseases, there are fewer patients," he noted. "Therefore the price per course of treatment is going to go up."

According to data from the Iqvia Institute, 72 percent of new drugs targeted orphan diseases—those affecting fewer than 200,000 Americans—in 2024, up from 51 percent in 2019. Over 40 percent of these treatments were for cancer. Others, like Bristol Myers' schizophrenia drug Cobenfy, priced at $22,500 per year, were aimed at larger populations.

The Pharmaceutical Research and Manufacturers of America defended the industry, saying list prices for rare disease drugs do not reflect the overall value these medicines provide, both in terms of health outcomes and system-wide savings.

The rise in prices also follows years of scientific progress. Since the human genome was mapped in 2003, researchers have gained better insight into the genetic roots of disease, leading to the development of more targeted therapies. Drugmakers also benefit from government incentives for rare disease treatments, including more extended market exclusivity.

Still, these drugs often come with staggering price tags. Zevra Therapeutics' Miplyffa, for example, was launched at over $1 million annually. Orchard Therapeutics' gene therapy Lenmeldy came to market at $4.25 million for a one-time dose — the most expensive treatment approved last year.

Of the 57 novel drugs cleared by the FDA in 2024, seven were cell or gene therapies. Despite the steep costs, companies argue that these treatments may reduce emergency room visits and hospital stays — and, in some cases, offer the promise of a cure.